Gene Therapy Offers Hope for Genetic Hearing Loss in Sweden
Significant advancements in gene therapy show promise for restoring hearing in patients with genetic disorders.
Key Points
- • All ten patients in the study experienced improved hearing and tolerated the treatment well.
- • Average sound perception improved from 106 decibels to 52 decibels post-treatment.
- • Lead researcher emphasized the significance of findings, particularly for adolescents and adults.
- • Future research aims to explore other genes responsible for hearing loss.
A groundbreaking study from researchers at Karolinska Institutet, published on July 2, 2025, has shown that gene therapy can significantly improve hearing in patients with genetic forms of deafness. The study involved ten patients aged 1 to 24 years, all suffering from hearing loss due to mutations in the OTOF gene, which plays a critical role in sound signal transmission from the ear to the brain.
The treatment utilized a synthetic adeno-associated virus (AAV) to deliver a functional OTOF gene directly into the inner ear through a single injection. Results were promising, with all patients experiencing enhanced hearing capabilities within a month post-treatment. Notably, the average sound level patients could perceive improved from 106 decibels to 52 decibels after six months, marking a significant recovery. A seven-year-old girl even achieved near-normal hearing levels, demonstrating the therapy's effectiveness particularly in younger patients.
Lead researcher Maoli Duan reflected on the importance of these findings, emphasizing that this is the first instance that the methodology has been tested on adolescents and adults, showing considerable enhancements in their quality of life. The treatment was generally safe, with only minor side effects observed, such as a temporary reduction in neutrophils. The team intends to continue research into other gene targets responsible for hearing loss, such as GJB2 and TMC1, which pose further challenges but are promising based on animal study results. The research was supported by various funding sources, including Otovia Therapeutics Inc. and several Chinese research programs.